CASE STUDY THERAPY AREA: ONCOLOGY EFFICIENT ENROLMENT AND DOSE ESCALATION PROCESS ENSURES RAPID ACCESS TO CRITICAL DATA
Page A of A
CASE STUDY AN OPEN-LABEL, MULTI-CENTRE, NON-RANDOMISED PHASE I DOSE-ESCALATION STUDY TO INVESTIGATE THE SAFETY AND TOLERABILITY OF (study drug) GIVEN AS MONOTHERAPY IN PATIENTS WITH RELAPSED/REFRACTORY NON-HODGKIN’S LYMPHOMA (NHL) AND RELAPSED/REFRACTORY CHRONIC LYMPHOCYTIC LEUKAEMIA (CLL).
SPONSOR OBJECTIVES: Our sponsor needed to ensure First in Patient study success in Europe, in a highly competitive oncology field amongst sites and patient availability. A minimum enrolment of 42 patients was required to complete the study. The multiple dose and ascending dose study planned for 6 cycles allowed patients to remain in the study until progression of the disease.
CHALLENGES OF THE STUDY • To investigate the safety and tolerability of escalating oral doses of (study drug) given as monotherapy in patients with relapsed/refractory NHL and relapsed/refractory CLL. • To examine peripheral blood lymphocytosis and recovery with increasing doses of (study drug) • To obtain preliminary data on the anti-tumour efficacy of (study drug) given as monotherapy in patients with relapsed/refractory NHL and relapsed/refractory CLL. • To investigate pharmacogenomic markers and their association with clinical response and toxicity.
PATIENT PROFILE • Patients with a confirmed diagnosis of and documented history of relapsed or refractory malignant disease (B-cell lymphoma and/or CLL ([CLL confirmed by NCI Working Group Criteria, 2008]) for which no therapy of curative or high priority exists.
Page 1 of 2
CASE STUDY STUDY CHALLENGES: Due to the highly competitive field, it was important to ensure that the sites were fully committed to the study and did not have any competing projects planned. The original plan was to enrol approx. 42 patients, but by the end of the study the actual number enrolled was 90 patients. This came about through changes in the study and decisions by the Sponsor to expand various cohorts. It was crucial that the study design fulfilled all scientific/regulatory objectives without being excessively complex as this could potentially discourage investigators and patients from taking part. Favourable Investigational Medicinal Product (IMP) results led to the potential for ‘competition’ among centres for slots in the dose escalation cohorts. Thus, it was critically important that data was provided to the safety committee in a timely and efficient manner ensuring that dose escalation decisions could proceed rapidly and in an unbiased way. Timely supply of the drug was necessary to ensure it was available at the time any new cohort was going to be enrolled at the correct dose, to prevent any delay in patient enrolment.
OUR SOLUTIONS: Working closely with the sponsor, we ensured that the final protocol was as ‘patient friendly’ as possible. Each potential site was visited by experienced Simbec-Orion medically qualified personnel and/or an oncology Project Manager to ensure they were fully committed and engaged with the project, this was key to keep momentum and enrolment of patients. Careful monitoring of patients enrolled by each site within each cohort to ensure that where multiple patients were available in any one site, then priority was given to sites that had not enrolled in that cohort. Thanks to close communication with the Sponsor, we could optimize IMP management efficiency ensuring cost effectiveness for IMP. When a decision was made to escalate the dose, an order for the next level was prepared rapidly - the study drug was available when required without storing excessive quantities at the sites.
OUTCOMES: The therapeutic principle was rapidly established, thanks to rapid and efficient conduct of the study. The sponsor was able to communicate the results to the scientific and business communities earlier than anticipated with a larger data set of 90 patients rather than 42 patients originally planned. This led to a highly favourable outcome for the sponsor in terms of the commercial development of the product.
Number of countries:
02
(UK & FRANCE)
Number of sites:
08
Number of enrolled patients:
90
Timelines FPI to Final CSR:
4.5 YEARS
Page 2 of 2
MISSION STATEMENT We are Simbec-Orion. An international, full service, boutique CRO; growing by bringing together the best possible people, healthcare professionals and drug developers, from all areas of clinical development. We focus on a defined series of core therapeutic areas, where we can make best use of our skills elegantly to design, execute and deliver our clients’ clinical development needs. We are making Simbec-Orion a highly respected and profitable boutique CRO. We do this by working for our clients with excellence, commitment and passion for our trade. We provide an environment in which our colleagues can continue to grow and develop. We will always remember that our work leads to the improvement of patients’ lives
To find out how Simbec-Orion can help with your clinical development program email info@SimbecOrionCRO.com Or call our business development team on +44 1753 578080 FRONT COVER IMAGE: HODGKIN LYMPHOMA CYTOLOGY
Simbec-Orion Group Limited 7 Bath Road, Slough Berkshire SL1 3UA United Kingdom